Twelve-year-old Kendric Cromer has spent the entirety of his young life battling sickle cell disease (SCD), a group of genetic blood disorders that affects hemoglobin, causing extreme bouts of pain crises that largely restrict one’s quality of life. After a long-anticipated rollout, Kendric has become the first person in the country to undergo the federally approved stem cell gene therapy designed to cure sickle cell disease.
Children’s National Hospital in Washington, D.C. is the first to conduct stem cell extraction for the commercially approved procedure by way of Bluebird Bio, a biotechnology company based out of Somerville, Massachusetts, that commercializes gene therapy for severe genetic diseases. Dr. Andrew Dale Campbell, director of the Children’s National Comprehensive Sickle Cell Disease Program, shared his team’s eagerness to see Kendric through this journey to a hopeful place of stability.
“It’s been very inspiring. We are excited because we draw from Kendric’s inspiration, we draw from his strength and also his family, who are a very strong support system [for him],” Campbell told the Informer. “We, the Children’s National team, initially did not know that we were going to be the first. We just put things in order [and] in motion to make sure that this was going to happen in a way that will allow one of the first patients to get started as soon as possible.”
According to the Centers for Disease Control and Prevention (CDC), roughly 100,000 people live with SCD across the United States, with an overwhelming 90% of those affected being African Americans.
Many people battling SCD experience difficulties accessing appropriate care despite their extensive health needs. They also deal with stigma and having their symptoms dismissed when they actually seek care.
Currently, Kendric has completed his first two stem cell extractions, now awaiting the next steps from Bluebird Bio to determine if the process can continue, or if more stem cell samples are needed before engineering the extracted cells.
Kendric’s mother, Deb Cromer, expressed her gratitude for the opportunity to potentially transition her son to a more stable, less painful state.
“We see the light at the end of the tunnel and know that our child is the first to experience it outside of research – to know that it’s safe and the doctors here believe in it,” Kendric’s mother told NBC News Washington. “I would go to the end of the earth to make sure he was cured. There’s nothing I wouldn’t do for my son, but this makes me proud.”
While optimism surrounds the inception of Bluebird Bio’s Lovo-Cel Gene Therapy, approved by the Federal Drug Administration (FDA) in December of 2023, medical experts do acknowledge the potential downsides of the cumbersome process, which in some cases can raise the risks of infertility, or risk of infection due to required chemotherapy during the process.
“Anytime you’re dealing with chemotherapy, versus fertility, [you run] the risk of not having the ability to have children, because chemo can have an adverse effect on the reproductive organs,” said Campbell. “So, the first thing is, you have to do testicular preservation or Oocyte retrieval. All of this is important moving forward if [patients] want to have their own children. Two, the chemotherapy can increase your risk of infection. But because it’s their own bone marrow in the process, they don’t have to take any rejection medications. That is minimized, [as] the anti-rejection medications put you at higher risk.”
But while the process presents potential for negative outcomes and unanswered questions of durability post procedure, a handful of successful clinical trials have encouraged Campbell to feel hopeful in the chance of patients responding positively to the ground-breaking procedure.
“I feel pretty confident because the great thing is we’ve had patients who have gone through it, and so far, in the clinical trial it has been very positive and the patients for the most part, have responded very well. Every year that goes by we’ve [reached] another year of confidence and hope. Nothing is 100% proof, but overall I feel pretty hopeful.”
Accessing Gene Therapy
Bluebird Bio’s life-changing gene therapy comes at a hefty expense to SCD patients, as the New York Times ranked the procedure as one of the most expensive medical treatments of its time at roughly $3.1 million. The treatment is so extensive that the company can only maintain the bandwidth to treat the cells of between 85 to 105 patients per year.
To date, about 20 patients were reportedly eligible and interested at Children’s National, but the decision of who receives treatment is largely based on which patient is most ill, along with whose insurance will cover the exorbitantly expensive costs.
“We’re proud that Children’s National was the first to initiate this process in terms of gene therapy, but[while] I can tell you that all of our patients are deserving, Kendric is [also] a great example of someone who wants a better quality of life in the foreseeable future,” Campbell explained. “He wants to achieve his goals without interruptions of hospitalizations and pain crises, so he’s very focused. A lot of our patients are very focused on what they want to do in life.”
